Bradley W G
Department of Neurology, University of Vermont College of Medicine, Burlington 05401.
Muscle Nerve. 1990 Sep;13(9):833-42. doi: 10.1002/mus.880130910.
The lack of effective therapy for many of the chronic neuromuscular diseases such as amyotrophic lateral sclerosis, hereditary motor sensory neuropathy (Charcot-Marie-Tooth disease), spinocerebellar degenerations and idiopathic polyneuropathy has led to a search for substances that may stimulate peripheral nerve regeneration. Two such agents that have been proposed are gangliosides (mixed purified bovine brain gangliosides, Cronassial) and thyrotropin releasing factor (TRH). Studies on both of these agents were initially reported with enthusiasm to be successful, but later double-blind controlled studies have failed to confirm these findings. This review provides critical analysis of the designs of studies of potentially effective agents in chronic neuromuscular diseases, and emphasizes the power of the placebo response, and the importance of designing placebos which are indistinguishable from the trial medication other than in the active effect.
对于许多慢性神经肌肉疾病，如肌萎缩侧索硬化症、遗传性运动感觉神经病（夏科-马里-图斯病）、脊髓小脑变性和特发性多发性神经病，缺乏有效的治疗方法，这促使人们寻找可能刺激周围神经再生的物质。两种被提出的此类药物是神经节苷脂（混合纯化牛脑神经节苷脂，Cronassial）和促甲状腺激素释放因子（TRH）。最初关于这两种药物的研究热情洋溢地报道称取得了成功，但后来的双盲对照研究未能证实这些发现。本综述对慢性神经肌肉疾病潜在有效药物的研究设计进行了批判性分析，并强调了安慰剂反应的影响力，以及设计出除了具有活性效果外与试验药物难以区分的安慰剂的重要性。
